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AX 4070

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Drug Profile

AX 4070

Alternative Names: AX-4070

Latest Information Update: 30 May 2025

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At a glance

  • Originator ProQR Therapeutics
  • Class Oligonucleotides
  • Mechanism of Action Gene expression modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Research CNS disorders

Most Recent Events

  • 20 Mar 2025 Early research in CNS disorders in Netherlands (Parenteral) (ProQR Therapeutics pipeline, March 2025)
  • 13 Mar 2025 ProQR Therapeutics has patent pending for Axiomer® program in multiple countries
  • 13 Mar 2025 ProQR Therapeutics has patent protection for Axiomer® program in Australia, Brazil, Europe,India, Japan, New Zealand, China, Canada, Israel, New Zealand, Japan, Russia, South Africa, South Korea, USA and Europe

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